According to a story from BioSpace, the biopharmaceutical company Sobi and the biotechnology company NovImmune SA have recently announced that the US Food and Drug Administration (FDA) has approved Gamifant,…
According to a story from the biotechnology company Amgen, a review from the European Medicines Agency (EMA) has recommended that the indication for blinatumomab, marketed as BLINCYTO®, be expanded. The…
According to the US Centers for Disease Control and Prevention, there have been 10 more confirmed cases of acute flaccid myelitis (AFM), a polio-like illness that mostly affects children. This now…
The Newborn Screening Saves Lives Act was originally introduced to facilitate the spreading of awareness and education related to newborn disease screenings. The screening of newborns has had a major…
According to a story from Scimex, the Children's Medical Research Institute in Sydney, Australia, has recently agreed to a new partnership with LogicBio Therapeutics, a biotechnology company from Boston, MA.…
We are entering the Thanksgiving season. I’ve been taught all my life that it shouldn’t be only a season, but an attitude year around. But how? By telling Mom thank…
According to a story from CheckOrphan, a wide array of experts are raising concerns about India's rare disease policy, which was first unveiled in May of 2017. Despite being hailed…
Glioma is a type of cancerous tumor that originates in the brain. It has various types and the tumors range in aggressiveness. There are around 11,000 people just in the…
According to a story from tonic.vice.com, Liz Aronin and Jamie Mills both thought that everything was going smoothly with their newborn son Quinn, but within a few months, it became…
99% Invisible recently reported on the history of "Orphan Drugs." These drugs are an enormous source of income for pharmaceutical companies - but the explosion in their production is a…
Polycythemia Vera or PV, is a rare blood cancer. It's caused by a mutation which results in the overproduction of red blood cells. Too many blood cells significantly increases your…
Cystic fibrosis patients in the UK are planning a protest on November 19th, 2018 with the goal of getting Orkambi, an important cystic fibrosis drug, covered on the NHS. This…
According to a story from ncbnewyork.com, a recent study found that $7 million on donations on crowdfunding sites for medical patients have gone towards treatments that are medically unsound and…
According to a story from Buzzfeed News, Paige Rogers was 21 years old when she was admitted to the hospital. She was having trouble breathing because of cystic fibrosis. Over…
Neuroendocrine tumors are growths which form from neuroendocrine cells in various places throughout the body. They can occur in the gastrointestinal track, the pancreas, the lungs, the thymus, and other…
According to a story from Raconteur, finding sufficient funding for the development of new therapies for rare diseases has always been a major challenge. Rare diseases are defined in the…
According to a story from UCI News, University of California Irvine scientists have developed an innovative immunotherapy that could screen patients in order to analyze the composition of their tumors…
The annual Global Genes Rare Patient Advocacy Summit is wonderful place for families affected by rare disease to come together and interact face to face with advocacy leaders in addition…
Some experiences are seen through rose-colored glasses while others are hazy or ambiguous. Many, we try desperately to remember while others we we wish we could forget. And a special…
I hope that you know a wonderful doctor. The type who listens wholly, respects you, validates your concerns and opinions, and goes above and beyond to make sure you get…
A recent study has looked into the use of video visits with patients by clinicians in Sweden, to try to find out how doctors decide which patients to offer them…
In the movie "Dallas Buyers Club" the main character, Ron Woodroof, contracts HIV/AIDs and starts getting drugs from an organization that imports medicine from Mexico. Similarly, many London patients are…
According to a story from Vietnam News, mother Huyen Trang has seen two of her children face death's door within mere months of being born. Her first child died at…
In 1993, researchers found one of the causes of familial amyotrophic lateral sclerosis (ALS). ALS is a neurodegenerative disorder which affects the motor neurons. Motor neurons are responsible for transmitting…
Happy Friday! Winter is coming and so is FDA news! Take a moment to check out some recent FDA announcements. FDA Approves Triplet Immunotherapy Combination for Multiple Myeloma Big news…
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